Improved Double-Nicking Strategies for COL7A1-Editing by Homologous Recombination

Current gene-editing approaches for treatment of recessive dystrophic epidermolysis bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low efficiencies and safety concerns that complicate implementation in clinical settings. We present a strategy for efficient and prec...

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Bibliographic Details
Main Authors: Thomas Kocher, Roland N. Wagner, Alfred Klausegger, Christina Guttmann-Gruber, Stefan Hainzl, Johann W. Bauer, Julia Reichelt, Ulrich Koller
Format: Article
Language:English
Published: Elsevier 2019-12-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119302586